NS-050/NCNP-03-101 in Boys With Duchenne Muscular Dystrophy (DMD)
Study Overview
- Age
- 4–15 years
- Phase
- Phase 1/2
- Sponsor
- NS Pharma
- Therapeutic Approach
- Exon Skipping
- Variant Requirement
- Confirmed DMD exon deletion in the dystrophin gene that is amenable to skipping of exon 50 to restore the dystrophin mRNA reading frame.
- Eligible Sex
- Male
- Ambulation
- Ambulatory
- Study Start (Actual)
- 2024-09-18
- Primary Completion (Estimated)
- 2028-03
- Study Completion (Estimated)
- 2028-03
- Enrollment (Estimated)
- 20
- Countries
- United StatesCanadaJapanSouth KoreaTurkiye
Study Requirements and Criteria
Steroid Use
Stable dose of glucocorticoid for at least 3 months and the dose is expected to remain on a stable dose for the duration of the study.
Inclusion Criteria
- Male ≥ 4 years and <16 years of age;
- Able to walk independently without assistive devices;
- Able to complete the TTSTAND without assistance in <20 seconds;
Exclusion Criteria
- Evidence of symptomatic cardiomyopathy;
- Current or previous treatment with anabolic steroids (e.g., oxendolone, oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug;
- Currently taking another investigational drug or has taken another investigational drug within 3 months prior to the first dose of study drug;
- Surgery within the 3 months prior to the first dose of study drug or planned during the study duration;
- Having taken any gene therapy.
- Other exclusion criteria may apply.
Contacts and Locations
Contacts and Locations
Contact
- Name: Trial info
- Phone Number: 1-866-677-6276
- Email: [email protected]
Locations
This study has 19 locations
United States
California Locations
Los Angeles, California, United States, 90027
Children's Hospital Los Angeles
Colorado Locations
Aurora, Colorado, United States, 80011
Children's Hospital Colorado
Illinois Locations
Chicago, Illinois, United States, 60611
Ann & Robert H. Lurie Children's Hospital of Chicago
Kansas Locations
Kansas City, Kansas, United States, 66103
University of Kansas Medical Center
Pennsylvania Locations
Philadelphia, Pennsylvania, United States, 19104
Children's Hospital of Philadelphia
Pittsburgh, Pennsylvania, United States, 15213
University of Pittsburgh Medical Center
Canada
Alberta Locations
Calgary, Alberta, Canada
Alberta Children's Hospital
British Columbia Locations
Vancouver, British Columbia, Canada
British Columbia Children's Hospital
Ontario Locations
London, Ontario, Canada
London Health Sciences Centre
Japan
Gifu Locations
Nagara, Gifu, Japan, 502-8558
National Hospital Organization Nagara Medical Center
Hyōgo Locations
Nishinomiya, Hyōgo, Japan, 663-8501
Hyogo Medical University Hospital
Miyagi Locations
Sendai, Miyagi, Japan, 989-3126
Miyagi Children's Hospital
Osaka Locations
Toyonaka, Osaka, Japan, 560-8552
NHO Osaka Toneyama Medical Center
Tokyo Locations
Kodaira, Tokyo, Japan, 187-8551
National Center of Neurology and Psychiatry
South Korea
Gyeongsangnam-do Locations
Yangsan, Gyeongsangnam-do, South Korea
Pusan National University Yangsan Hospital
Seoul, South Korea
Seoul National University Hospital
Turkey (Türkiye)
Ankara, Turkey (Türkiye)
Ankara Bilkent City Hospital
Istanbul, Turkey (Türkiye)
Istanbul University
Istanbul, Turkey (Türkiye)
Yeditepe University Kosuyolu Hospital
Clinical Trial Registry
NCT ID
NCT06053814This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.